A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II

Phase 2

Recruiting

• Conditions: Hunter syndrome; Mucopolysaccharidosis Type II (MPS II); 10029305

• Registration Number: NL-OMON53946
• Lead Sponsor: Denali Therapeutics Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 9 April 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Not specified
• Target Recruitment: 3

Inclusion Criteria

1. Participants aged >=2 to <6 years (Cohort A) or >=6 to <17 years (Cohort
B).
2. Confirmed diagnosis of MPS II (for Cohort A, nMPS II; for Cohort B, nnMPS
II).
3. For non-run-in Cohort A and Cohort B only: Be on maintenance enzyme
replacement therapy (ERT) and have tolerated a minimum of 4 months (ie, 16
weeks) of idursulfase therapy during the period immediately prior to screening.

Exclusion Criteria

1. Have a documented pathogenic or likely pathogenic variants that are known to
cause developmental delay or decline, cognitive dysfunction, seizures, or other
significant CNS disorders

2. Previously received an IDS gene therapy or stem cell therapy

3. Received any CNS-targeted MPS ERT within 6 months prior to screening

4. Have a contraindication for lumbar punctures and/or magnetic resonance
imaging (MRIs)

5. Participated in any other investigational drug study or used an
investigational drug within 60 days prior to screening or intend to receive
another investigational drug during the study

Study & Design

• Study Type: Interventional
• Study Design: Not specified